Biological and clinical insights from a randomized phase 2 study of an anti-oncostatin M monoclonal antibody in systemic sclerosis.

Objectives: The cytokine oncostatin M (OSM) is implicated in the pathology of SSc. Inhibiting OSM signalling using GSK2330811 (an anti-OSM monoclonal antibody) in patients with SSc has the potential to slow or stop the disease process.

Methods: This multicentre, randomized, double-blind, placebo-controlled study enrolled participants ≥18 years of age with active dcSSc.

Conditional assurance: the answer to the questions that should be asked within drug development.

In this paper, we extend the use of assurance for a single study to explore how meeting a study's pre-defined success criteria could update our beliefs about the true treatment effect and impact the assurance of subsequent studies. This concept of conditional assurance, the assurance of a subsequent study conditional on success in an initial study, can be used assess the de-risking potential of the study requiring immediate investment, to ensure it provides value within the overall development plan. If the planned study does not discharge sufficient later phase risk, alternative designs and/or success criteria should be explored.

Safety and efficacy of ozanezumab in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled, phase 2 trial.

Background: Neurite outgrowth inhibitor A (Nogo-A) is thought to have a role in the pathophysiology of amyotrophic lateral sclerosis (ALS). A monoclonal antibody against Nogo-A showed a positive effect in the SOD1 mouse model of ALS, and a humanised form of this antibody (ozanezumab) was well tolerated in a first-in-human trial. Therefore, we aimed to assess the safety and efficacy of ozanezumab in patients with ALS.